News

A new extended release formulation of zilucoplan, a potential self-administered therapy for patients with generalized myasthenia gravis (gMG), given once weekly, achieved complement 5 (C5) inhibition similarly to zilucoplan administered daily, pre-clinical data in non-human primates show. Zilucoplan, developed by Ra Pharmaceuticals, is an artificial peptide that binds to…

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Almost 90% of patients diagnosed with myasthenia gravis develop other diseases, or comorbidities, while about 70% have treatment-related complications, a review study from India reports. These additional disorders may contribute to a poorer outcome for these people, making early recognition and management of comorbidities essential for improving patient care and overall…

A combination therapy of high-dose cholinesterase inhibitors, such as Mestinon, with calcineurin inhibitors, such as Prograf, increases the chances of muscle cramps in patients with Myasthenia Gravis, thereby reducing their quality of life, a Japanese study has found. The study, “Treatment of Myasthenia Gravis…

Researchers have identified five candidate therapies for treating myasthenia gravis (MG) by using a new computational model that also predicts key biological processes involved in the disease. The study, “Building the drug-GO function network to screen significant candidate drugs…

Ra Pharmaceuticals is preparing to launch a Phase 3 clinical trial to assess the therapeutic potential of zilucoplan for the treatment of patients with generalized myasthenia gravis (gMG). This comes after the company’s completion of End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA). The 12-week study — a single, pivotal,…

Robotic surgical removal of the thymus seems to be safe and effective and may reduce or eliminate the need for steroid therapy in myasthenia gravis (MG) patients older than 60 at onset. The study, “Results of robotic thymectomy performed in myasthenia gravis patients older than 60 years at…

Alexion Pharmaceuticals‘ Soliris (eculizumab) significantly reduces fatigue perceived by patients with generalized myasthenia gravis, a study finds. The study, “Eculizumab improves fatigue in refractory generalized myasthenia gravis,” was published in Quality of Life Research. Myasthenia gravis (MG) is an autoimmune disease caused…

For more than four decades, comedian Jerry Lewis hosted the MDA Labor Day Telethon on behalf of the Muscular Dystrophy Association. That annual event helped the MDA become the nation’s largest non-government source of funding for neuromuscular disease research, with more than $1.4 billion disbursed since its establishment 68…

A recent report described a rare case of a woman presenting with a sudden decrease in voice volume who was ultimately diagnosed and successfully treated for myasthenia gravis (MG). The case study, “Hypophonia as only presenting symptom in myasthenia gravis — a diagnostic dilemma in poor…